Company Description (as filed with the SEC)
We are a development-stage specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting (rare) orphan neuromuscular and neurological diseases and disorders. We currently have three pharmaceutical products in development: • Firdapse™. In October 2012, we licensed the North American rights to Firdapse™, a proprietary form of amifampridine phosphate, or chemically known as 3,4-diaminopyridine phosphate, from BioMarin Pharmaceutical Inc. (BioMarin). As part of our agreements with BioMarin, we are currently taking over the sponsorship of their ongoing Phase III clinical trial evaluating Firdapse for the treatment of Lambert-Eaton Myasthenic Syndrome, or LEMS, a rare and sometimes fatal autoimmune disease characterized by muscle weakness. ... More ...